As I posted recently, Asterias Biotherapeutics has had good early safety results so far in its historic stem cell trial for spinal cord injury and now the company presented some early, encouraging hints at efficacy. However, a caveat here is that this is not an RCT (randomized controlled trial).

Ed Wirth, CMO of Asterias, presented the early data at the 55th Annual Scientific Meeting of the International Spinal Cord Society. You can take a look at the very interesting talk slides here. Asterias is a subsidiary of BioTime.

According to the press release(PR) from the company, while only 4 of 5 patients are 90 days or more after treatment, all patients have shown some improvement in motor function and a subset have a substantial improvement, meeting the target at this point. This trial is funded in part by CIRM so kudos to our state’s stem cell agency on this.

MIT Tech Review is on the story to for more background. I’ve also posted from Dr. Wirth’s PowerPoint the study design image below.

Bottom line. Overall, I think this development is encouraging. While still early in the clinical trial and drug testing process, the fact that there is a good safety profile and early indications of possible efficacy yield real hope. Still, it is important to be cautious in interpreting early data. We’ll all need to follow this trial and then look for future RCT results to clarify how promising this drug is overall in the long haul.

I’ve posted the key bullet points from the Asterias PR below the fold.

At Day 90 of follow up, 4 of 4 patients dosed have improved one motor level on at least one side, 2 of 4 patients have improved two motor levels on at least one side, and 1 patient has improved two motor levels on both sides.

The average UEMS improvement at Day 90 for the 4 patients that have reached this follow up was 9.5 points.

The results to date from Cohort 2 show no serious adverse events related to AST-OPC1, the injection procedure, or immunosuppression with low-dose tacrolimus. In addition, data from the study indicate that AST-OPC1 can be safely administered to patients in the subacute period after severe cervical spinal cord injury.

The average UEMS improvement for the 3 patients was 5.0 points at Day 90, and they continued to improve an average of 7.0 points at 1 year.

Despite the very low dose used in this safety cohort, at 1 year of follow up 1 patient in Cohort 1 has improved one motor level on one side and 2 patients have improved one motor level on both sides.

The 12-month results from Cohort 1 show no serious adverse events related to AST-OPC1, the injection procedure, or immunosuppression with low-dose tacrolimus. In addition, data from the study indicate that AST-OPC1 can be safely administered to patients in the subacute period after severe cervical spinal cord injury.”